The first healthy volunteer has been enrolled in a Phase 1 study testing ABX1100 — a novel substrate reduction therapy from Aro Biotherapeutics — as a treatment for Pompe disease. This will be the first-in-human trial for the experimental therapy, meaning it’s the first time ABX1100 has been…
Phase 1 trial
Single and multiple ascending doses of MZE001, Maze Therapeutics‘ experimental oral substrate reduction treatment for Pompe disease, were well tolerated and reduced glycogen levels in healthy volunteers in a Phase 1 trial, data show. These findings support a Phase 2 trial of MZE001 in people with Pompe disease,…
MZE001, an experimental oral substrate reduction treatment for Pompe disease, was well-tolerated in both single and multiple doses in a study conducted in healthy volunteers, according to Maze Therapeutics, the therapy’s developer. Based on the trial’s findings, Maze is planning to launch a new study in 2023 to…
Duke Health researchers are planning to launch a Phase 1 trial to test the safety of a new investigational gene therapy they developed to treat patients with Pompe disease. They are currently screening (NCT03285126) adults with late-onset Pompe disease to select eligible participants for the upcoming trial.